Total number of Results for Transgenic mouse model are 15
ASPsiRNA ID Pairing of target region (top) and ASP-siRNA (bottom) Efficacy (mut)(%) Efficacy (wt) (%) Diff Gene Disease Mutation RefSNP Analysis Pubmed ID
ASPsiRNA ID Mutant allele/ASP-siRNA Efficacy
for mutant(%)
Efficacy
for wild (%)
Relative Difference (%) Gene Disease Mutation RefSNP Analysis Pubmed ID
aspsirna1001 GGAGACUUGCGCAAUGUGA
CCUCUGAACGCGUUACACU
80 20 60 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna0170 UGAAGUGAAUCUGGAUGCAG
ACUUCACUUAGACCUACGUC
60 10 50 APP Alzheimers disease c.2010_2011delGAinsTC (p.Lys670_Met671delinsAsnLeu) rs28186516
19532137
aspsirna0342 GACAAAGAUGCUGUGGCCGAU
CUGUUUCUACGACACCGGCUA
74 10 64 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.281G>C,p.Gly94Ala rs12191243
16857362
aspsirna0804 CGAUUCCAUGUUCAUGAGU
GCUAAGGUACAAGUACUCA
80 74 6 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.281G>C,p.Gly94Ala rs12191243
15768029
aspsirna0805 UCGAUUCCAUGUUCAUGAG
AGCUAAGGUACAAGUACUC
50 42 8 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.281G>C,p.Gly94Ala rs12191243
15768029
aspsirna1000 UGGAGACUUGCGCAAUGUG
ACCUCUGAACGCGUUACAC
95 10 85 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna1002 GAGACUUGCGCAAUGUGAC
CUCUGAACGCGUUACACUG
96 23 73 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna1003 UGGAGACUUGGGCAAUGUG
ACCUCUGAACCCGUUACAC
82 5 77 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna1004 GGAGACUUGGGCAAUGUGA
CCUCUGAACCCGUUACACU
20 46 -26 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna1005 GAGACUUGGGCAAUGUGAC
CUCUGAACCCGUUACACUG
NA NA NA SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
12934714
aspsirna3121 GAUCAAGACCCUCAAAAAC
CUAGUUCUGGGAGUUUUUG
56 42 14 KRT6A Pachyonychia congenita (PC) c.513C>A,p.Asn171Lys rs59685571
19935778
aspsirna3122 CCCUCAAAAACAAGUUUGC
GGGAGUUUUUGUUCAAACG
35 0 35 KRT6A Pachyonychia congenita (PC) c.513C>A,p.Asn171Lys rs59685571
19935778
aspsirna3178 UUGCCAGGUUGCAGCUGCUCUU
AACGGUCCAACGUCGACGAGAA
90 26 64 HTT Huntington disease (HD) rs362306 rs362306
25849618
aspsirna3258 GGCCUCUUGGCUCUUGGAU
CCGGAGAACCGAGAACCUA
80 2 78 KRT9 Epidermolytic palmoplantar keratoderma (EPPK) c.500delAinsGGCT,p.Tyr167delinsTrpLeu rs59317143
27003758
aspsirna3259 GGCCUCUUGGCUCUUGGAU
CCGGAGAACCGAGAACCUA
73 10 63 KRT9 Epidermolytic palmoplantar keratoderma (EPPK) c.500delAinsGGCT,p.Tyr167delinsTrpLeu rs59317143
27003758